Redesigning the Clinical Effectiveness Research Paradigm Innovation and Practice-Based Approaches: Workshop Summary (2010) / Chapter Skim
Currently Skimming:
1 Evidence Development for Healthcare Decisions: Improving Timeliness, Reliability, and Efficiency
1 Evidence Development for Healthcare Decisions: Improving Timeliness, Reliability, and Efficiency
Pages 51-86
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 51... ...
Information systems are capturing much larger quantities of data at the point of care; new techniques are being tested and used to analyze these rich datasets and to develop insights on what works for whom; and research networks are being used to streamline clinical trials and conduct studies previously not feasible. An examination of how these innovations might be used to improve understanding of clinical effectiveness of healthcare interventions is central to the Roundtable on Value & Science-Driven Health Care's aim to help transform how evidence is developed and used to improve health and health care.
|
From page 52... ...
The need to find new approaches to accelerate the development of clinical evidence and to improve its applicability drove discussion at the Roundtable's workshop on December 12–13, 2007, Redesigning the Clinical Effectiveness Research Paradigm. The issues motivating the meeting's discussions are noted in Box 1-1, the first of which is the need for a deeper and broader evidence base for improved clinical decision making.
|
From page 53... ...
The development in recent years of a broad range of clinical research approaches, along with the identification of problems in generalizing research results to populations broader than those enrolled in tightly controlled trials, as well as the impressive advances in the potential availability of data through expanded use of electronic health records, have all
|
From page 54... ...
. Table 1-1 provides brief descriptions of the many approaches to clinical effectiveness research discussed during the workshop -- and these methods can be generally characterized as either experimental or non-experimental.
|
From page 55... ...
When feasible and appropriate, trials are "double blind" -- i.e., patients and trialists are unaware of treatment assignment throughout the study. Pragmatic Experimental design that is a subset Primary, Required Clinical Trial of RCTs because certain criteria are may include (PCT)
|
From page 56... ...
N of 1 trial Experimental design in which an Primary Required individual is repeatedly switched between two regimens. The sequence of treatment periods is typically determined randomly and there is formal assessment of treatment response.
|
From page 57... ...
For example, if the disease is rare, or when researchers would like to observe the natural history of a disease or real world practice patterns. Ecological Non-experimental design in which the Primary or No Study unit of observation is the population secondary or community and that looks for associations between disease occurrence and exposure to known or suspected causes.
|
From page 58... ...
tiveness, feedback is crucial on how well new products and interventions work in broad patient populations, including who those populations are and under what circumstances they are treated. Redesigning the Clinical Effectiveness Research Paradigm Growing opportunities for practice-based clinical research are presented by work to develop information systems and data repositories that enable greater learning from practice.
|
From page 59... ...
Physiology-based modeling has been used to enhance and extend existing clinical trials, to validate RCT results, and to conduct virtual comparative effectiveness trials. In part, this is a taxonomy and classification challenge.
|
From page 60... ...
President, Institute of Medicine An increasingly important focus of the clinical effectiveness research paradigm is the efficient development of relevant and reliable information on what works best for individual patients. A brief look at the past, present, and future of clinical effectiveness research establishes some informative touchstones on the development and evolution of the current research paradigm, as well as on how new approaches and directions might dramatically improve our ability to generate insights into what works in a clinical context.
|
From page 61... ...
, focused on agricultural comparisons but articulated many of the critical principles in the design of controlled trials that are a hallmark of current clinical trials. It would not be until after World War II, that the first clinical trial on a medical intervention would be recorded.
|
From page 62... ...
Unfortunately, despite the huge number of randomized trials being conducted a number of needs are not being met by the current "randomized trial only" strategy. Effectiveness Research to Inform Clinical Decision Making Archie Cochrane, the inspiration for the Cochrane Collaboration, posed three deceptively simple yet critical questions for assessing clinical evidence.
|
From page 63... ...
or organization of service, institutional performance, and the patient's role. Thinking critically about what is being evaluated and mapping what is appropriate, effective, and efficient, for the various types of questions, is an ongoing and important challenge in clinical effectiveness research.
|
From page 64... ...
These perspectives all extend differences to what kind of method will be suitable for what kind of question in what kind of circumstance. The questions of what it is we are evaluating, for whom, and with whose perspective and purpose in mind are decidedly nontrivial, and in fact they can be important guides as we reflect on the strengths and weaknesses of a variety of approaches and formulate better approaches to clinical effectiveness research.
|
From page 65... ...
In particular, discerning meaningfully relevant information in the health context begs for closer attention, and strategies for innovative approaches to data mining are emerging -- strategies analogous, if you will, to the way that Internet search engines apply some order to the vast disarray of undifferentiated information spread across the terabyte-laden database of the World Wide Web. We are also seeing the development of innovative trial and study methodologies that aim to compensate for some of the weaknesses of clinical trials with respect to external validity.
|
From page 66... ...
As we consider the array of methods and develop strategies for their use in clinical effectiveness research, a guiding notion might be "a metaexperimental strategy" that aids the determination of which new methods and approaches to learning what works, and for what specific purposes,
|
From page 67... ...
Agency for Healthcare Research and Quality All of us share a common passion for developing better evidence so we can improve health care, improve the value of health care, and provide clinicians, patients, and other relevant parties with better information to make health decisions. In that context, this paper explores a central question: How can our approach to clinical effectiveness research take better advantage of emerging tools and study designs to address such challenges as generalizability, heterogeneity of treatment effects, multiple co-morbidities, and translating evidence into practice?
|
From page 68... ...
If there is a lesson there, it is that evidence that we are developing has to be valid, broadly available, and relevant. Traditional Hierarchies of Evidence: Randomized Controlled Trials Traditional hierarchies of evidence have by definition placed the randomized controlled trial (RCT)
|
From page 69... ...
In short, nonrandomized studies can enrich our understanding of how patient treatments in practice differ from those in trials. A good case in point are two studies published in the 1980s by the Lipid Research Clinics showing that treatment to lower cholesterol can reduce the risk of coronary heart disease in middle-aged men (The Lipid Research Clinics Coronary Primary Prevention Trial Results.
|
From page 70... ...
Nonrandomized trials also help researchers to pursue the similarities and differences between the trial population and the typical target population, and between trial intervention and typical interventions. Nonrandomized trials enable researchers to examine the heterogeneity of treatment effects in a patient population that in some ways or for some components may not look very much like the trial population.
|
From page 71... ...
Clearly, external validity becomes a problem or a challenge, and that dilemma has been the subject of many lively debates. As noted by Nicholas Longford, "clinical trials are good experiments but poor surveys." In a paper a few years ago in the Milbank Quarterly, Richard Kravitz and colleagues suggested that the distribution of specific aspects and treatment effects in any particular trial for approval could result in a very different sense of the expected treatment effect in broader populations (Kravitz et al., 2004)
|
From page 72... ...
Subgroup analyses are not reported regularly enough for individual patient metaanalyses. Moreover, we need to look beyond RCTs to inform judgments about the applicability and heterogeneity of treatment effects.
|
From page 73... ...
versus explanatory clinical trials and how PCTs might move the field closer to the notion of embedding research into care delivery and contending directly with issues confronting clinicians. In PCTs, hypotheses and study design are formulated based on information needed to make a clinical decision; explanatory trials are designed to better understand how and why an intervention works.
|
From page 74... ...
Since then, however, the literature seems to have gone silent on such issues. It would be a useful activity to revisit these questions and actually develop an inventory of what we know about practical clinical trials and how difficult they can be.
|
From page 75... ...
Observational Studies This leads to the question of whether observational studies can reduce the need for randomized trials. Clearly observational studies are a preferred alternative when clinical trials are impractical, not possible, or unethical (we seem to debate that question a lot even though we are not particularly clear about what it means)
|
From page 76... ...
Similarly, observational studies that meet the criteria shown in the figure (and perhaps others as well) , and which are done very well, might in some instances be considered better evidence than a randomized trial, if a randomized trial is poorly done.
|
From page 77... ...
These papers are a valuable new resource for scientists who are committed to advancing the comparative effectiveness and safety research, and this is an area in which AHRQ intends to continue to push.2 Approaches to Turning Evidence Into Action The Agency for Healthcare Research and Quality has several programs directed at turning evidence into action. AHRQ's program on comparative effectiveness was authorized by Congress as part of the Medicare Modernization Act and funded through an appropriation starting in 2005.
|
From page 78... ...
Center activities also focus on decision support and other approaches to getting information to the point of care for clinicians, as well as on making information relevant and useful to patients and consumers. The Eisenberg Center is developing two new translational guides, the Guide to Comparative Effectiveness Reviews and Effectiveness and OffLabel Use of Recombinant Factor VIIa.
|
From page 79... ...
, using electronic health record data from 8 organizations representing more than 200 clinicians and over 350,000 patients (Agency for Healthcare Research and Quality, 2008a)
|
From page 80... ...
These efforts underscore the commitment by many in the research community to creating better data and linking those data with better methods to translate them into more effective health care. Health Care in the 21st Century We must make sure that we do not lose sight of the importance of translating evidence into practice.
|
From page 81... ...
5,000 20% 21% 22% 23% 24% 25% 26% 27% 28% 29% 30% 31% 32% 33% 34% 35% Relative Risk Reduction in Mor tality from Drug Therapy FIGURE 1-5 Potential lives saved through quality improvement -- The "break-even point" for a drug that reduces mortality by Figure 1-5.eps 20 percent. SOURCE: Woolf, S
|
From page 82... ...
T1, T2, and T3 represent the three major translational steps in the proposed framework to trans step test the discoveries of prior research activities in progressively form the healthcare system. The activities in each translational landscape broader settings to advance discoveries originating in basic science research through clinical research and eventually to widespread implementation through transformation of healthcare delivery.
|
From page 83... ...
FIGURE 1-7 Evolution of translational research.
|
From page 84... ...
In this ideal system, actionable information would be available -- to clinicians and patients -- "just in time," and evidence would be continually refined as a by-product of healthcare delivery. The goal is not producing better evidence for its own sake, although the challenges and debates about how to do that are sufficiently invigorating on their own that we can almost forget what the real goals are.
|
From page 85... ...
2007. Creating and synthesizing evidence with decision makers in mind: Integrat ing evidence from clinical trials and other study designs.
|
From page 86... ...
2007. Developments in post-marketing comparative effectiveness research.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.